Mesoblast
chief executive
Silviu Itescu
will ask US regulators if the company can lodge scaled-back data from ­clinical trials in order to speed up final approval for its novel stem cell therapies for diseases such as heart failure and cancer.

In 2014, Mesoblast intends to run four phase three clinical trials, which is the final stage before regulatory approval. It will study the effect of its adult stem cell therapies on congestive heart failure, back pain caused by intervertebral disc disease, auto-immune condition Crohn’s disease and graft versus host disease (a common side effect of bone marrow transplants to treat cancer).

For heart failure, Mesoblast, in partnership with Israeli drug company Teva Pharmaceutical, will measure whether the injection of stem cells in patients who have had a heart attack prolongs the time until the occurrence of another cardiac event that leads to death. The inherent risk with this study design is that if the stem cells are successful, it might be a long time before patients die and Mesoblast can report final data comparing treatment with control groups. This delays its regulatory applications, which further extends the time to making sales.

Mr Itescu said Mesoblast intended to report an interim end point of the trial, based on that with current therapies, 20 per cent of heart failure patients have a cardiac event in the first 12 months after a heart attack.

“That’s the basis of why 1700 patients are being recruited to be able to show a statistical difference between the 20 per cent event rate in controls and say a 10 or 15 per cent rate in our treated population," he said.

“For that to be meaningful and significant, you need about 800 patients per arm [placebo v treatment]."

‘Breakthrough designation’ requested

Mesoblast would also manage this challenge by asking the US Food and Drug Administration to give it “breakthrough designation" for all its phase three trials.

Related Quotes

Company Profile

This status is for therapies of major diseases without suitable ­therapies.

“Almost every one of the indications [diseases] we’re going after are sizeable diseases with major unmet needs where our technology seems to have a unique mode of action and opportunity," Mr Itescu said. “That lends itself now to the discussions we can have with the regulators around accelerating and shortening potentially the phase-three programs."

Canaccord analyst Matthijs Smith said the breakthrough status was only introduced in mid-2012 and only finalised for products like Mesoblast’s in June 2013.

Although some drugs have been given the status it is too early to know how this would affect the approval process, Dr Smith said.

“One would expect if an expedited process leads to approval, it is equally likely to be a requirement on the company to provide ongoing monitoring to ensure the process hasn’t blocked any data that we have to know about."

Mesoblast investors have had a rocky ride over the past 12 months, with the stock peaking at $7.49 in February after new patents were granted in the US and China. The shares closed down 3.4 per cent to $5.70 on Wednesday. The stock has mostly bounced between $5.40 and $6.30 in the past year.

Dr Smith said the volatility has “not necessarily been linked to news flow but where it is linked, the gains have been rapidly returned".

Phase three trials looking at the effect of adult stem cells in the treatment of Crohn’s disease and graft ­versus host disease are under way or have begun patient enrolment.

Mr Itescu said Mesoblast would announce results from a phase two trial on back pain in “the next couple of weeks", which would hopefully lead to a phase three trial.